P23: UFO: Utility-Based Curve-Free Phase I/II Clinical Trial Design Identifying the Optimal Dose of Immunotherapies
Conference: ASA Biopharmaceutical Section Regulatory-Industry Statistics Workshop 2023
09/29/2023: 9:45 AM - 10:30 AM EDT
Posters
Room: White Flint Foyer
Immunotherapy is a cutting-edge treatment that utilizes the patient's immune system to combat cancer. However, current clinical trial designs for immunotherapy rely on intricate parametric models that are challenging to validate in practice and interpret clinically, hindering the translation of statistical design into practical trial implementation and producing undesirable outcomes. To address these issues, we propose the UFO design, a curve-free phase I/II clinical trial design that incorporates the distinct characteristics of immunotherapy to identify the optimal biological dose (OBD) that maximizes the risk-benefit trade-off for patients. The UFO design dynamically determines the dose escalation and de-escalation by simultaneously considering immune response, toxicity, and efficacy, allocating patients to the dose with the highest desirability in the risk-benefit tradeoff. Unlike most existing designs, the UFO design does not rely on any parametric model assumptions and produces favorable results under any clinically significant dose-response curves. Additionally, we have extended the UFO design to handle delayed outcomes. Our comprehensive simulation studies demonstrate that the UFO design produces favorable operating characteristics.
Phase I/II clinical trials
Immune response
Immunotherapy
Risk–benefit tradeoff
Delayed outcomes
Presenting Author
Yingjie Qiu, Indiana University
CoAuthor(s)
Yong Zang, Indiana University
Yi Zhao, Indiana University
Topic Description
Clinical Trial Design (e.g., Innovative/Complex Design, Estimands, Master Protocol)
ASA Biopharmaceutical Section Regulatory-Industry Statistics Workshop 2023
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